What does the drug do?
Tegoprubart is an antibody against a protein called CD40 ligand (CD40L) which is present on the surface of some immune cells. Antibodies are proteins that bind to and block a particular target. CD40L plays a role in regulating the immune response and that can trigger inflammation in the spinal cord. Research has shown that the CD40L pathway is overactive in people with MND. By inhibiting CD40L, AT-1501 could block or delay the activation of the damaging inflammatory immune response which could delay the onset or slow the progression of MND and increase survival.
The results of the phase 2 clinical trial found tegoprubart to be safe and well-tolerated in people living with ALS. They also found that the drug hit the target pathways within the body, with some ALS biomarkers (biological signals of the disease) being reduced. You can read more here.
Previous Clinical Trials
The Phase 1 trial enrolled 8 participants and its primary outcome was safety and tolerability. The trial showed that all participants tolerated all dose levels. The pharmacokinetics (how it is processed in the body) of the drug was found to be as the researchers predicted. You can read more about the results of the trial here.
The Phase 2a trial recruited 54 MND patients to take part in the open-label, multidose study in the US. Participants received one of three increasing intravenous doses of AT-1501 once every two weeks for 11 weeks. Investigators continued to monitor them for safety and tolerability for another eight weeks (19 weeks in total). You can find out more about the trial here.
The topline results of the trial found that tegoprubart was well-tolerated. The trial also found that the drug demonstrated dose dependent target engagement and inflammatory biomarkers associated with ALS were observed and reduced.
Last updated: 21/12/2022